GETTYIMAGES, CRISTIAN BAITG (TARGET AND EYE); ISTOCKPHOTO.COM, PIAL HOVIK (DNA HOOP)
The concept is simple: if a mutated gene is causing a problem, replace or supplement it with a new, accurate copy. In theory, such a strategy could not just treat, but cure countless human genetic diseases. In practice, however, developing safe and effective gene therapies has not been easy. Even when identifying a disorder’s genetic basis is fairly straightforward, finding the appropriate delivery vector to target the diseased tissues in the body, while avoiding unintended consequences, has challenged would-be gene therapists for more than 20 years. But more and more researchers are convinced that the technique is on the brink of becoming a common medical practice.
“It’s an incredibly exciting time for the field,” says researcher and medical oncologist David Kirn, founder, president, and chief ...
